Clinical Trial: NU-0129 in Treating Patients With Recurrent Glioblastoma or Gliosarcoma Undergoing Surgery

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: A Phase 0 First-In-Human Study Using NU-0129: A Spherical Nucleic Acid (SNA) Gold Nanoparticle Targeting BCL2L12 in Recurrent Glioblastoma Multiforme or Gliosarcoma Patien

Brief Summary: The purpose of this research study is to evaluate the safety of the study drug, NU-0129, based on Spherical Nucleic Acid (SNA) platform when infused in patients with recurrent glioblastoma multiforme or gliosarcoma. The SNA consists of nucleic acids arranged on the surface of a small spherical gold nanoparticle. This is a first-in-human trial to determine the safety of NU-0129. NU-0129 can cross the blood brain barrier (a filtering mechanism that carry blood to the brain). Once within the tumor, the nucleic acid component is able to target a gene called Bcl2L12 that is present in glioblastoma multiforme, and is associated with tumor growth. This gene prevents tumor cells from apoptosis, which is the process of programmed cell death, thus promoting tumor growth. Researchers think that targeting the Bcl2L12 gene with NU-0129 will help stop cancer cells from growing.

Detailed Summary:

PRIMARY OBJECTIVES:

I. To assess the safety of intravenous NU-0129 in patients with recurrent glioblastoma multiforme (GBM) or gliosarcoma (GS).

SECONDARY OBJECTIVES:

I. To analyze drug concentration in serum at specific time points after drug administration.

II. To demonstrate intratumoral penetration of NU-0129. III. To assess the feasibility of giving NU-0129 as a standard treatment for recurrent GBM or GS.

TERTIARY OBJECTIVES:

I. To analyze tumor tissue for Bcl2L12 expression levels after NU-0129 administration.

II. Preliminary response (progression free survival [PFS] and overall survival [OS] at 6 months; overall response rate [ORR]).

OUTLINE:

Patients receive NU-0129 intravenously (IV) over 20-50 minutes and undergo standard of care tumor resection within 8-48 hours.

After completion of study treatment, patients are followed up at 7, 14, 21, and 28 days and then every 84 days for up to 2 years.


Sponsor: Northwestern University

Current Primary Outcome: Incidence of Adverse Events [ Time Frame: Up to 28 days after study drug administration ]

To evaluate the safety of intravenous NU-0129 in patients with recurrent GBM or GS, the number of adverse events will be assessed and will be graded according to the NCI's Common Terminology Criteria in Adverse Events (CTCAE) version 4.03.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Drug concentration in blood after drug administration [ Time Frame: At 1, 3, 5, 10, 30, and 60 minutes, and 4, 8, and 24 hours post infusion ]
    Blood samples will be collected post-infusion to analyze drug concentration at specific time points after drug administration.
  • Biodistribution of NU-0129 in tumor tissue [ Time Frame: Up to 2 years ]
    Tissue will be collected during the scheduled surgery and assayed with Inductively Coupled Plasma Mass Spectrometry (ICPMS) to analyze the concentration of particles in various parts of tumor tissue.
  • Feasibility of giving NU-0129 as a standard treatment [ Time Frame: Up to 2 years ]
    Feasibility will be calculated as the rate of successful production, delivery, and administration of the investigational product and subsequent resection.


Original Secondary Outcome: Same as current

Information By: Northwestern University

Dates:
Date Received: January 11, 2017
Date Started: May 4, 2017
Date Completion: July 2022
Last Updated: May 16, 2017
Last Verified: May 2017